UPFRONT

Serendipity

Jason S. Slakter, MD

The process of bringing a new drug or therapeutic approach from initial concept to regulatory approval and eventual delivery to the patient is a difficult and complex one, particularly for individuals or small start-up companies. To be successful, it takes four key components: a good idea; a good team; money; and serendipity.

The first step is having a good concept, which may begin with the identification of an unmet medical need, a careful and thorough search for a therapeutic strategy, and diligent laboratory experimentation to develop the therapy. Alternately, unique properties of an existing molecule or device may be discovered that lend themselves to other disease states. In either case, you need a good team of people with the needed skills (scientific, administrative, business development) and enthusiasm to carry the project forward.

This brings us to the third point: the need for money. In the early stages, financing may come from grants or support from universities. In other situations, small companies may seek out "friends and family" as angel investors to cover expenses. If the preclinical data are positive, money from venture capitalists may then help drive the next stages of development. To fund the more expensive clinical trials needed for regulatory approval, attention turns to larger venture firms or partnerships with pharmaceutical companies.

The final and perhaps most important component is often a simple matter of timing and luck. In preparing for the recent American Academy of Ophthalmology meeting, I had an opportunity to experience the role serendipity plays in drug development. An initial evaluation of the outcome data from a 24-month phase 2 trial for dry AMD was discouraging. The primary endpoint of the trial had not been met, and there were discussions as to whether to abandon the project.

Fortunately, however, the scientific team conducted a thorough analysis of the study details and discovered there were issues related to drug manufacturing that impacted the data. They also revealed more exciting findings: the data pointed to a potential biomarker for use in future clinical studies, and the drug appeared to have an effect on reducing the development of choroidal neovascularization. Clearly, we will have to await the results of large-scale clinical investigations for proof of efficacy for this drug. However, these new discoveries may not have occurred had it not been for the need to understand the initially disappointing outcome.

Without question, the process of developing new drugs and therapies is an intricate and complicated one and something that is critical for our ability to better help our patients. To provide our readers with more information on this subject, Retinal Physician recently initiated a new column called "Innovation in Retina," which debuted in the September issue and will begin its regular run in January. In addition, we plan to devote an entire afternoon at Retinal Physician Symposium 2011 to the cycle of drug and therapeutic development. We hope this will provide you an opportunity to learn more about these issues and become more involved in the process. Ultimately, we all stand to benefit.